Agenda Details

Learn more about sessions and presentations at the 26th Annual Meeting

Detailed Program

Accelerate your drug development program in rare disease with robust natural history data: an evolving space

Session Details

Co-Chair

Kathleen M. Kirby. , Barnstable, MA

Natural History Studies - Introduction

Kathleen M. Kirby, BS. Viridian, Barnstable, MA

How to design an NH study that regulators and clinicians support

Adam Shaywitz, MD, PhD. BridgeBio Pharma, Palo Alto, CA

Designing a development plan for a robust control data source

John Balser, PhD. Veristat LLC, MA

Listening to the public/patients/caregivers

Cara O'Neill, MD. Cure Sanfilippo Foundation, SC

Running NH studies in a robust and verifiable way

Anne Ruggles. Pariser, MD. Alltrna, Bethesda, MD

Panelist

Cara O'Neill, MD. Cure Sanfilippo Foundation, SC

Panelist

Kristen Pappas. , CA

Panelist

Anne Ruggles. Pariser, MD. Alltrna, Bethesda, MD

Panelist

Adam Shaywitz, MD PhD. BridgeBio Pharma, Palo Alto, CA

Panelist

John Balser. , MA

Panelist

Daniel Chung, DO, MA. SparingVision, Philadelphia, PA

Key take-aways; Future directions

Samantha Parker. Innoskel, Biot, France

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